Access to medicines still a problem for rare disease patients

Budget pressures and bureaucracy among obstacles

Patients with rare diseases are not getting the drugs they need because of budget pressures, bureaucratic obstacles and problems of scale, panellists told a Friends of Europe ‘Café Crossfire’ lunch debate on Wednesday 21 June. Speakers at the event called for more collaboration between doctors, industry, insurers, patients and EU governments to solve this issue.

Around 30 million Europeans suffer from rare diseases like cystic fibrosis or Duchenne muscular dystrophy. And while there are more than 6,000 known rare diseases, according to the Orphanet database, therapies exist for only 400 of them. “Clearly this is an area where there is a need for critical mass,” said Xavier Prats Monné, European Commission Director-General for Health and Food Safety. “Patients are the ones we should be thinking of.”

The Commission has already introduced incentives for pharmaceutical companies to develop new drugs, which has increased the number of drugs to treat rare diseases from eight to more than130 in the last 15 years. It has also set up a series of European Reference Networks, bringing patients and specialists together to help speed up and aid treatment. But more ‘soft incentives’ are needed to encourage the use of data, increase neonatal screening and train doctors to diagnose rare illnesses.